Tom Curran, PhD, FRS, and researchers at the Center for Childhood Cancer Research are investigating the effects of drugs that inhibit the sonic hedgehog cell signaling pathway on the growth of pediatric medulloblastoma.
Genetic analysis revealed that approximately 30percent of all pediatric medulloblastomas have an activated sonic hedgehog pathway. This finding led to the identification of a small molecule inhibitor using mouse medulloblastoma models developed in this laboratory.
Subsequent Phase I human clinical trials (and more recently Phase II trials) revealed that medulloblastoma tumors containing sonic hedgehog activation mutations could be successfully treated and eliminated using the small molecule inhibitor.
Currently, efforts are underway to develop predictive diagnostic tests to determine whether or not pediatric patients with medulloblastoma may respond to targeted sonic hedgehog inhibitor treatment before surgery, radiation or conventional chemotherapy is initiated.